What question did this study set out to answer?

To evaluate the safety and efficacy of GD2-specific CAR T-cell therapy for treating refractory retinoblastoma.

April 13, 2026Open Access

Intravitreal GD2‐Specific Chimeric Antigen Receptor T‐Cell Therapy for Refractory Retinoblastoma

Key Points

To evaluate the safety and efficacy of GD2-specific CAR T-cell therapy for treating refractory retinoblastoma.
Administered two intravitreal injections of GD2-CAR T cells under compassionate use
Monitored patient for localized ocular inflammation and intraocular pressure
Conducted histopathological analysis of tumor tissue
Localized ocular inflammation developed post-injection but subsided
Increased intraocular pressure led to enucleation within 6 weeks
Histopathology revealed dense lymphocytic infiltration around the tumor

Abstract

ABSTRACT Effective treatments for advanced, treatment‐resistant retinoblastoma (RB) remain limited. GD2‐specific chimeric antigen receptor (CAR) T cells show potent antitumor activity with minimal toxicity but have not previously been evaluated in RB. Under compassionate use, a patient with refractory RB received two intravitreal GD2‐CAR T‐cell injections. Localized ocular inflammation developed around the tumor within 1 week, subsided, and recurred after the second injection. Elevated intraocular pressure required enucleation at 6 weeks. Histopathology demonstrated dense peritumoral lymphocytic infiltration without anterior segment involvement. Intravitreal GD2‐CAR T‐cell therapy was feasible, well tolerated, and induced localized immune activation and tumor regression, supporting further investigation.

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